MENLO PARK, Calif.--(BUSINESS WIRE)--PellePharm, a clinical-stage biopharmaceutical company committed to
developing patidegib, a topical hedgehog inhibitor to treat basal cell
carcinomas (BCCs), including those in Gorlin Syndrome, a devastating
orphan disease, announced its launch with financing from BridgeBio
Pharma. This financing supports the development of topical patidegib
through the completion of two phase 2 clinical trials that are underway,
including a clinical trial in Gorlin Syndrome that just completed
enrollment. In addition, the company has a drawable pool of capital from
BridgeBio Pharma that enables it to finance future clinical trials
through registration.

PellePharm was founded by a team of globally recognized experts in
dermatology, oncology and hedgehog signaling and is managed by a team of
industry experts. The company is dedicated to finding an effective
therapy to manage the extensive tumor burden in Gorlin Syndrome, a rare
genetic disease that causes patients to develop multiple BCCs. Gorlin
Syndrome affects about 10,000 people in the United States and 15,000
people in the European Union. The disease results from a mutation in a
tumor-suppressor gene, which acts as the primary inhibitor of the
hedgehog signaling pathway. PellePharm’s founders developed a topical
gel formulation of a proprietary hedgehog inhibitor that it exclusively
licensed from Infinity Pharmaceuticals to help mitigate certain adverse
events observed with oral hedgehog inhibitors.

“It made sense that a potent inhibitor of the hedgehog pathway should
provide a therapeutic benefit for patients suffering from Gorlin
Syndrome, but we needed an approach that would allow us to target the
disease at its source without eliciting harmful toxicity reactions,”
said Ervin Epstein, M.D., co-founder of PellePharm. “We are hopeful that
patidegib will provide the balance of targeted treatment without the
adverse events associated with oral formulations.”

PellePharm’s approach attracted the attention of industry veterans Mark
de Souza, Ph.D. and Karl Beutner, M.D., Ph.D. who joined the team. “It’s
not often that you find a program as attractive as PellePharm’s
patidegib,” said de Souza, executive chairman of PellePharm. “This
investigational treatment has the potential to address a very high unmet
need among patients with Gorlin Syndrome, whose current standard of care
is to undergo multiple surgeries each year to manage their symptoms. We
know that hedgehog pathway inhibitors have therapeutic potential for
this condition, and we believe PellePharm’s topical formulation may
offer a truly effective treatment option for these patients.”

PellePharm is currently conducting two double-blinded,
placebo-controlled, randomized, phase 2 clinical trials that are
evaluating the safety, tolerability and effect of topical patidegib on
both pre-existing tumors and the frequency of the development of new
tumors. A U.K.-based study is evaluating 18 patients with Gorlin
syndrome to determine whether the product shrinks the tumors present at
the beginning of the study and reduces the number of new tumors that
develop during the trial. A U.S.-based study is evaluating 36 patients
with sporadic BCCs to determine whether tumor diameter decreases after
12 weeks of treatment. Both studies also will evaluate safety and
tolerability, as well as a biomarker (GLI-1) of hedgehog signaling.
Enrollment in the U.K. trial is complete and topline data is expected in
the second quarter of 2017.

“PellePharm is really at the intersection of precision oncology and
monogenic dermatology - these are the types of assets we look for at
BridgeBio Pharma,” said Frank McCormick, investment committee member of
BridgeBio Pharma and board member of PellePharm. “Gorlin Syndrome is a
well-researched disease that can be treated directly at its source. We
have had the privilege of knowing the founding and operating team of
PellePharm for years and look forward to working with them to bring
patidegib to patients in need.”

Founders and Scientific Advisory Board MembersPellePharm
was founded by a group of globally recognized experts in dermatology,
oncology and hedgehog signaling who continue to lend expertise and
counsel to the PellePharm management team. Founders and scientific
advisory board members include:

About Basal Cell Carcinomas (BCCs) and Gorlin SyndromeBasal
cell carcinomas (BCCs) are a type of skin cancer that begins when one of
the skin cells spontaneously develops a mutation in its DNA, usually due
to UV radiation (sunlight, tanning lamps). They commonly occur in
sun-exposed areas like the face and neck and generally in people of
European descent with lighter skin and in the elderly and in people with
chronic sun exposure. There are more than four million BCCs diagnosed in
the United States each year. BCCs are usually treated with surgery,
which can leave disfiguring scars.

Gorlin Syndrome, also known as nevoid basal cell carcinoma syndrome, is
a rare genetic disease where patients develop many BCCs. Patients with
Gorlin Syndrome have heritable mutations in the tumor suppressor gene
encoding Patched1 (PTCH1), which acts as the primary inhibitor of the
hedgehog signaling pathway; this leads to hundreds of BCCs, especially
on the face and sun-exposed areas. The standard of care is surgery, as
there are no FDA-approved drugs for Gorlin Syndrome. Individuals who
have severe Gorlin Syndrome have as many as 30 surgeries per year, many
of which can be scarring.

About PellePharmPellePharm is developing patidegib, a novel
hedgehog pathway inhibitor, to treat basal cell carcinomas (BCCs),
including those in Gorlin Syndrome, a devastating rare genetic skin
disease. The company is focused on developing and commercializing this
treatment to reduce the burden of disease and improve quality of life by
potentially reducing invasive and painful surgeries and the scarring
that often accompanies them. Topical patidegib is a first-in-class
topical gel formulation of a proprietary hedgehog inhibitor exclusively
licensed from Infinity Pharmaceuticals. Patidegib is currently being
studied in two phase 2 clinical trials for the treatment of BCCs and
Gorlin Syndrome.

About BridgeBio PharmaBridgeBio Pharma develops medicines
for genetic diseases with significant unmet need. The company is
primarily focused on pre-commercial products, utilizing its advantages
in sourcing and operating to find and build value at each stage of
development. Founded in 2015, and backed by KKR and Perceptive Life
Sciences, BridgeBio Pharma is actively developing multiple clinical and
pre-clinical programs.